title: Missing data issues at the FDA Center for Biologics Evaluation and Research.
abstract: Missing data in clinical trials pose a tremendous problem in the regulatory setting. The U.S. Food and Drug Administration relies on statistically rigorous evidence of safety and efficacy as a precondition for granting marketing approval for drugs, biologics, and many devices. The presence of missing data can compromise the integrity of any statistical analysis, further complicating a regulatory decision-making process with significant public health, legal, and financial implications. We review the unique missing data challenges faced by the Center for Biologics Evaluation and Research and discuss limitations in our current approach to these issues and potential directions for improvement.
authors.affiliation: Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, Maryland, USA. John.Scott@fda.hhs.gov
authors.forename: John A
authors.initials: JA
authors.lastname: Scott
authors.forename: Henry
authors.initials: H
authors.lastname: Hsu
mesh_terms: D001688:Biological Products / Q000009:adverse effects* / Q000627:therapeutic use*; D002986:Clinical Trials as Topic; D003627:Data Interpretation, Statistical; D017277:Drug Approval / Q000331:legislation & jurisprudence; D023381:Endpoint Determination; D033161:Government Regulation; D017408:Guidelines as Topic; D006801:Humans; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D001688:Biological Products